NHS refuses to pay for treatment of tumour that trials showed cut deaths by 25%

Dozens of families are clashing with the NHS over its refusal to fund treatments for a devastating children’s cancer that are not available in Britain.

At least 45 families with a child suffering from neuroblastoma, the biggest child killer after accidents, have public fundraising appeals under way. Many want to go to the United States, where a three-drug treatment is available that was shown in a trial published in a leading medical journal to cut deaths by 25%. But European regulators are still assessing the efficacy of the drugs.

The government’s cancer tsar, Prof Sir Mike Richards, said it would be wrong for the NHS to recommend that patients travel abroad to receive the treatment. He acknowledged the considerable concern from very well-informed parents as well as from charities, an MP and a high court judge. But he added: “I have to go with the advice from experts that I am given unless I think they have got it wrong.”

In Britain, some children with the disease, but not all, can join a European trial that will give them the key drug in the combination, a monoclonal antibody, either on its own or with one of the others.

John Rogers, a physicist whose four-year-old daughter, Stella, is now in remission with no sign of cancer after treatment costing £210,000 in Philadelphia, said: “None of the children are getting the treatment [in Britain] that is the best available treatment. They are using them as lab rats.”

Joining the trial is the only way children in Britain who have relapsed can get treatment on the NHS because the drugs are still experimental. Nice, the National Institute for Health and Clinical Excellence, often described as the NHS’s rationing body, is not involved because it can only appraise and recommend proven, licensed drugs.

Some families have been quietly advised by their NHS doctors to go to the US, Rogers claims. “The doctors are in such a bad position from what’s happened that they have shut the door and said if you can, you should go to the US. That’s a terrible position for a doctor to be in,” he said.

Richards is advised by the country’s leading experts involved in the European trial, including Dr Penelope Brock, the senior British expert who leads the collaboration running it.

Richards said neuroblastoma was complex. While everyone was agreed that the antibody, known as Ch14.18, was of benefit, it was uncertain whether the other drugs, called cytokines, were needed, given that they have side-effects.

British doctors believe they are doing their best for the entire population of children with neuroblastoma by running a trial with strict criteria for entry designed to answer specific questions about the effectiveness and safety of giving an antibody and a cytokine to these children.

But parents of children who are offered nothing are devastated when told there is nothing more that can be done.

Others, who are offered a trial place, reason that their child has only one chance and want the full drug treatment that has been shown to work in the US. All of them know children in the UK who have died – it is a relatively small community where families share each others’ harrowing experiences of trying and often failing to fight the cancer.

Questions about the European trial were raised in parliament last month by the Tory MP Andrew Selous. The parents of one child assigned randomly in the trial to receive just the single drug in the UK went to the high court, where a judge ruled it was in her best interests to receive both drugs.

Other parents, in desperation at coming to the end of their options in the UK, have travelled to Mexico, where clinics offer unproven treatments using sound waves to activate oxygen-releasing drugs.

The Downie family from Aberdeenshire raised money to take their daughter Olivia to Tijuana, where she became so ill she had to be transferred to a medical hospital and put on a life-support machine. They were then forced to appeal again for money to bring her home in an air ambulance. She died 48 hours later.

The government believes the NHS is doing all it should for the 100 children a year diagnosed with neuroblastoma. Richards cited the decision to send children for proton beam radiation therapy to the US at NHS expense: “Where we have good evidence that treatments abroad which can’t be delivered in this country would benefit children, we do pay.”

A second trial starting will allow children who are not currently eligible to be able to have antibody treatment without going abroad, but Richards said: “There will still be the question of ‘antibody plus’ which is what the trial is designed to test. We are still in a state of equipoise amongst the clinical community about the benefits, which is why we believe the clinical trial is the best approach and why, frankly, we can’t recommend patients to go abroad to get that treatment because we do not know that it is going to be of benefit to them and that the benefit will outweigh the harms.”

He added: “As yet, I have heard no compelling case that tells me they have got this wrong. I’m always prepared to relook at the situation and that may be what we need to do but, at the same time, I have not been given the evidence that would make me think this is something we should be doing when the clinical experts are not saying that to me.”

He accepted that more could be done to try to explain the government’s view to parents.

and said he would be happy to meet the charity and the clinical experts.